Gene Therapy for Dermatoses Using Epidermal Vectors

Award Information
Agency: Department of Health and Human Services
Branch: N/A
Contract: N/A
Agency Tracking Number: 21949
Amount: $50,000.00
Phase: Phase I
Program: SBIR
Awards Year: 1993
Solicitation Year: N/A
Solicitation Topic Code: N/A
Solicitation Number: N/A
Small Business Information
8080 N. Stadium Drive, Suite 2, Houston, TX, 77054
HUBZone Owned: N
Woman Owned: N
Socially and Economically Disadvantaged: N
Principal Investigator
 Fred D. Ledley
 (713) 795-0105
Business Contact
Phone: () -
Research Institution
This is part of a program to develop gene medicines for treatment of dermatoses such as psoriasis. We will utilize a novel DNA expression vector (HK1) capable of directing high-level tissue-specific expression from keratinocytes in the epidermis. The HK1 vector contains transcription and translation control elements derived from the human K1 gene. In transgenic animals, this vector directs high levels of gene expression specifically in proliferative basal cells as well as differentiated supra-basal cells of the epidermis. Phase-I studies will focus on demonstrating the feasibility of expressing therapeutic products in normal and hyperplastic skin after direct administration of the HK1 vector into the epidermis by particle bombardment. We will study expression of a marker gene beta-galactosidase as well as TGF-beta, a growth regulating factor which may be useful in inhibiting hyperproliferation in diseases such as psoriasis. We will study gene expression in normal mice and in the transgenic HK-1-TGF-alpha mouse where genetically engineered overexpression of TGF-alpha induces thickening, hyperproliferation, and hyperkeratosis of the skin similar to that seen in psoriasis. Phase-II studies will focus on developing novel methods for gene delivery to skin, expression in human epidermal models, and the development of new gene medicines for treating the hyperproliferation and inflammation characteristic of psoriasis.

* Information listed above is at the time of submission. *

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